Natural History vs. Registry Studies in Rare Disease

Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical studies and achieve meaningful outcome measures, researchers must apply known etiology and thorough knowledge of the disease’s progression. For rare diseases, this information is often minimal at best: patient numbers are small and historical data is spread across treating physicians who operate around the world.

It’s essential that pharma companies, patient advocates, and other stakeholders use data on the natural history of these diseases to drive discussion and formulate drug development strategy. There are two prevalent data collection approaches: registry studies, which may include broad collection of defined data, and natural history studies, used for controlled, detailed collection of data that is subject to regulatory scrutiny.

Key elements of this webinar will include:

• The importance of natural history information in rare disease research and the regulatory importance of comprehensive disease data
• The difference between registry and natural history studies, and how to decide which will better meet your needs
• How to design and conduct a registry or natural history study, and what resources are available to help you proceed

Speakers

Angi Robinson, Executive Director, Pediatrics & Rare Disease, Premier Research

Angi Robinson has been studying rare diseases at Premier Research for 14 years. She has provided oversight and management support for more than 30 rare disease studies in the U.S. and globally and has supported FDA pre-IND meetings, IND submissions, and NDA/BLA project directorship. Angi has worked on industry sponsored studies to support the approvals of four drugs with orphan drug designations.

Ms. Robinson helped launch the first Best Pharmaceuticals for Children Act coordinating center, collaborating with leaders at the National Institute of Child Health and Human Development. She served as Project Manager for highly complex trials, including sedation in pediatric intensive care units and pediatric bipolar disorder. Her experience includes multiple study designs including PK/PD, adaptive design, and FDA fast track and breakthrough therapy designations.

Ms. Robinson began her career in laboratory quality control and has worked in clinical trial management as a Project Director/Manager, Clinical Lead, and Clinical Monitor. She holds a bachelor’s degree in cell and molecular biology from Tulane University.

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Juliet Moritz, Executive Director, Rare Disease & General Medicine, Premier Research

Juliet Moritz has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare diseases, supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions.

Prior to joining Premier Research, Ms. Moritz was Associate Director of Global Project Management at PPD Inc., overseeing infectious and respiratory disease research, and prior to that was Associate Director of Clinical Research at Knopp Biosciences. She held senior positions at Wyeth, Theravance, and PPD and began her career as a clinical research associate.

Ms. Moritz holds a Master of Public Health degree from the Drexel University School of Public Health and a bachelor’s degree in biology from the University of Pennsylvania. She is a member of the National Organization for Rare Disorders and the Drug Information Association.

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